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gene editing

Back from summer vacation, Nathan and Laura are smoking hot as they look back over some exciting headlines.

The summer boiled over with plenty to talk about, but it was just this week that delivered most of the news for our discussion today. Novartis’ gene therapy based on CAR-T technology was approved Wednesday, making it the first gene therapy to be approved ever in the US. Analysts will be trying to figure out how high high is when it comes to the price tag, but Nathan and Laura explain why this therapy is a big deal for patients.

As for the first gene editing of embryos in the US that happened earlier in August? Nathan says, yes, it’s a first, but the big story is how "strikingly reliable the CRISPR edit is in germline vs the rest of the body."

Finally, we heard a few days ago that genetic testing provider, Invitae (recently featured here on the program) had sent out a large batch of false negative tests. Laura, a genetic counselor, says that in the absence of FDA regulation the system is operating on trust.

“And I want to say,” she adds, “ I trust Invitae. They’re a good lab, and I think they’re handling this well.”

The challenge for the first ever in-human gene editing trial, according to today’s guest, is with the delivery to the body.

“At the moment, the easiest place to deliver your gene or genome editing is to the liver, using AAV which are viruses that seek out and go to the liver cells," says Sandy Macrae, the CEO of Sangamo Therapeutics.

Sangamo is known for two things: They have pioneered the commercialization of an older gene editing technology called Zinc Fingers. And they have done a lot of work in the area of HIV.

Today, Sangamo is enrolling patients in a new trial which they say will be the first "in-vivo" trial using their Zinc Fingers for patients with hemophilia B, Hunter syndrome, and Hurler syndrome. The former gene editing work with the T cells of HIV patients, Sandy says, was done “ex-vivo”, or outside the body.

So why is Sangamo still using Zinc Fingers in the age of CRISPR? Sandy says that the older technology is much better developed for medical applications and is safer. The company has been able to get their off target effects to below the level of detection.

“When I was doing my postdoc, I would have used CRISPR. It’s better if you’re just wanting an easy experiment that isn’t about making a medicine but just getting a quick answer,” he says.

Because Sangamo has been the sole commercial developer of Zinc Fingers with not a lot of intellectual property dispute, the technology didn’t make the big PR splash that CRISPR has—nor, at the same time, did it generate all the fear.

We finish the interview with a question about what was the result of all Sangamo's work in HIV over the years.

It’s the end of the month--and the half year mark--so we open up today's monthly discussion with Nathan and Laura to include some of the headlines we’ve missed this year.

Last month a paper was published warning about the off target effects when using CRISPR. Laura and Nathan agree the kerfuffle which exploded into this month was more about Wall Street than adding anything new to science.

Remember the technology we used before CRISPR? Sangamo Biosciences launched the first ever “in vivo” (in human) trial for gene editing using Zinc Finger technology.

Also last month, the FDA approved a drug based on the genetic makeup of a tumor and not its location in the body. Nathan says, “Nyeh . . . And? Doctors have been doing it off label for years.” But Laura thinks it's more that just a symbolic gesture.

She also picked for her paper of the month this new study out by Robert Green’s group asking whether the benefits outweigh the risks for whole genome sequencing in healthy individuals.

"It’s preliminary, but it’s not PR. This is one of the really important questions for genomics in our time," says Laura.

Nathan highlights a paper propounding a new model for biology, the omnigenic model. Find out what that is in another fun and informative look back on the genomics headlines with Nathan Pearson and Laura Hercher.

Journalists listen to others telling them what actually happened all year long. But for this one week at the first of the year, we like to make up our own stuff.

January - The general mood at the annual J.P. Morgan Healthcare conference in San Francisco is one of relief.

“Last month the FBI caught the lead mastermind behind the pharma industry’s high drug prices, and he’ll be brought to justice,” says the CEO of a pharma giant to a room full of investors and journalists at the historic St. Francis Hotel. “Problem solved.”

Janitors have had a terribly busy time this week cleaning up all those jaws that were dropped on floors of research labs everywhere around the country.

Have you heard about this latest sexism scandal?

Two female co-authors of a scientific paper submitted their work to PLOS -- you know, the open access journal. You won’t believe what they heard back from the lone peer reviewer. They were told to go find “one or two male biologists” to be co-authors on the paper to increase its chances of being published.

Ouch!!! That hurts. Not only the co-authors but the rest of us.

Well, hold on, it gets worse. This chauvinist reviewer went on to say that “it might well be that on average men publish in better journals . . . perhaps simply because men, perhaps, on average, work more hours per week than women, due to marginally better health and stamina.”

What, a marginal ouch? Better health and stamina?

Then the two female co-authors decided to stop playing that video game, got their scarves, and went across the street to a cafe and ordered each a double latte.

Right? I mean, what’s the name of the video game, Doing Science Circa 1850?

"No," the lead female author says sitting down to her double latte. "The game is called, Anti-Civilization; Hang Out with a Primitive Tribe in Africa."

Now, last week we reported how former Congressman Newt Gingrich is calling for the doubling of the NIH Budget. Well this week, the bipartisan 21st Century Cures Initiative jumped in the game, calling for an increase to the NIH budget. $1.5 billion per year over the next five years. Well, we still like Newt’s suggestion. It’s bold. And let’s be positive. Let’s say we get the budget doubled. Now we got the problem of spending it. Right? Be careful what you wish for.

Since we’re absolutely sure that the NIH will come to Mendelspod for suggestions on how to spend the increase, we thought we should at least start getting prepared.

So we went online and posted a chat asking for suggestions from researchers how to spend the additional funds.

Would you like to hear a few of the responses?

Garbage In, Garbage Out - obviously the commentors are using pseudonyms -- from Phoenix, AZ, writes: “Write off half of it to waste. Because that’s the way it is. Over 50% of scientific research is non-reproducible.”

OK. We’re being taken seriously here.

Live to One Thousand from Cambridge, England, writes: “Spend all of the additional funding on aging research. We’ve tried the sniper method. Let’s just move in the troops.”

Wow, this is a serious chat. But hold on, a third person, Don’t Leave us Behind, out of San Diego, CA, writes:

Are you sure, Live to One Thousand? Aging research? I think we should take the additional $30 billion and fund Alzheimer’s research. You see, Nature is now asking us, are you sure you want to live longer?

Oh, and there’s one more here that just came in. It’s the author of the book called, From Buddhism to Big Dataism: Keeping up with the Newest Religions. And this author writes: "You might as well write the check now, NIH. Just make it out to the newest God on the Block, Big Data.

These online chats. They’re just too serious.

Now with all this talk of increasing the research budget, Francis Collins, the Director at NIH, immediately put out a notice about what he won’t fund. OK, he’s showing congress that he’s a good accountant. So what will the NIH not fund? Editing the human germline in embryos.

Now this is the only one he’s announced so far, but we heard that there are more. Did you want to hear about a couple?

OK, here’s one. This is something that the NIH will just not fund, no matter what. The proposal came in to do brain scans of all the presidential contenders and make the data openly available online for all the voters to see.

And here’s a whole category of projects to study why people are gay. What’s wrong with that? It turns out a Supreme Court Justice told the NIH that such studies are a gross overreach of the executive branch. That the direction of American society should not be up to scientists, but instead up to nine aging lawyers. "Besides," this justice said to the NIH, "we have the better costumes. White lab coats? Ha!"